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The 2020 Nobel Prize in Chemistry went to Emmanuelle Charpentier and Jennifer A. Doudna “for the development of a method for genome editing”. That method, formally known as CRISPR-Cas9 gene editing but often simply called CRISPR, allows scientists to precisely cut any strand of DNA they want. In the 8 years since its inception, CRISPR has been a boon to biologists, who have published thousands of studies showing that the tool can alter DNA in organisms throughout the tree of life, including butterflies, mushrooms, tomatoes and even humans.
“The number of discoveries in biomedicine that have had the impact that Jennifer and Emmanuelle had can be counted on the fingers of one hand: recombinant DNA, PCR [polymerase chain reaction], DNA sequencing and now CRISPR, ”says Fyodor Urnov, a gene-editing scientist at the University of California, Berkeley. “We have never had a technology as powerful and versatile as CRISPR genome editing.”
Charpentier, who is now in the Max Planck Unit for Pathogen Science, and Doudna, at UC Berkeley, began working together in 2011. The two scientists were inspired by a little-studied bacterial immune system that uses an enzyme called Cas9 to bite even the genes of invading viruses, which are saved as molecular mugshots. In 2011, Charpentier worked out the details of how a pair of bacterial RNA molecules controlled this process (Nature 2011, DOI: 10.1038 / nature09886).
The two scientists began to think about how they could transform this viral defense system into an easily programmable gene-editing tool. They synthesized a new molecule, called single guide RNA, that combines key features of the two bacterial RNAs and instructs Cas9 to cut a specific site on DNA (Sciences 2012, DOI: 10.1126 / science.1225829).
It was the tool scientists were waiting for. CRISPR is cheaper, faster, and easier to use than previous gene editing tools. Academic laboratories quickly adopted the technique. Today, scientists can order custom Cas9 and guide RNAs with the click of a button. CRISPR opened gene editing to the masses.
Charpentier, Doudna, and many others have founded biotech companies to apply CRISPR to agriculture and medicine. Some companies have already started testing experimental therapies that use CRISPR to treat, and potentially cure, rare genetic diseases and cancer in humans.
“It’s wonderful to see Jennifer and Emmanuelle recognized in this way,” says David Liu, a CRISPR scientist at the Broad Institute of MIT and Harvard. “With new treatments for human genetic diseases already in patients, with early positive results, the era of human genome editing has already begun, and Emmanuelle and Jennifer are two of the key pioneers responsible for ushering in this new era.”
